
Hereditary transthyretin amyloidosis is a complex, progressive, and potentially fatal genetic disorder caused by mutations in the TTR gene, leading to the accumulation of amyloid deposits in various organs, with significant impact on patient quality of life. However, recent advancements in diagnostic approaches and therapeutic options provide new hope for improved management and outcomes.


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The heterogeneity of hereditary amyloidosis can delay diagnosis and seriously affect prognosis and patient quality of life; these five things are need-to-know.

Hereditary Amyloidosis: 5 Things to Know

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Hereditary Transthyretin Amyloidosis: Challenges, Advancements, and Improving Patient Outcomes


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Hereditary transthyretin (ATTR) amyloidosis is a life-threatening autosomal dominant disease caused by misfolded transthyretin protein deposition, leading to neuropathy and cardiomyopathy. Early diagnosis and genetic testing are crucial, as new treatments targeting protein misfolding and gene silencing have shown promising results.


Morie Gertz, MD, reviews hereditary amyloidogenic transthyretin (ATTR) amyloidosis diagnosis and treatment.

Q&A: An Update on Hereditary Transthyretin Amyloidosis

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Comprehensive Overview of Hereditary Transthyretin Amyloidosis: Symptoms, Diagnosis, and Emerging Treatments
